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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene


NCTID NCT06291935 (View at clinicaltrials.gov)
Description (Show More)
Development Status Active
Indication Retinitis Pigmentosa
Disease Ontology Term DOID:110007
Compound Name VG901
Compound Description AAV2.NN-CNGA1
Sponsor ViGeneron GmbH
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant CNGA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2024-02-01
Completion Date 2026-04
Last Update 2025-03-28

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations Germany

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed 4/10/24

Resources/Links